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Donidalorsen Treatment in Children With Hereditary Angioedema
Donidalorsen Treatment in Children With Hereditary Angioedema

NCT07298447

RecruitingPhase 3

Sponsor: Ionis Pharmaceuticals, Inc.

Conditions: Hereditary Angioedema (HAE)

Interventions: Donidalorsen

Countries: United States, Italy, Poland, Spain

The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of donidalorsen in pediatric participants with hereditary angioedema (HAE) Type I (HAE-1) or Type II (HAE-2).

Eligibility overview

Sex: ALL

Age: 2 Years to 11 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
Key Inclusion Criteria:

1. Must be between the ages of 2 and less than 12 years, inclusive, at the time of informed consent and, as applicable, assent.
2. Must weigh at least 9 kg at the time of informed consent and, as applicable, assent.
3. Documented diagnosis of HAE-1/HAE-2 based upon both of the following:

   1. Documented clinical history consistent with HAE (SC or mucosal, non-pruritic swelling episodes without accompanying urticaria).
   2. Diagnostic testing results that confirm HAE-1/HAE-2: C1-inhibitor (C1-INH) functional level \<50% normal level AND complement factor C4 level below the lower limit of normal (LLN); OR a known pathogenic mutation in the SERPING1 gene.

Key Exclusion Criteria:

1. Must not have any screening laboratory abnormalities or any other clinically significant abnormalities during screening that would render a participant unsuitable for inclusion.
2. Must not have been treated with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer.
3. Concurrent diagnosis of any other type of recurrent angioedema, including idiopathic angioedema or HAE with normal C1-INH (HAE-nC1-INH or Type III).

Note: Other protocol-specified inclusion/exclusion criteria may apply.
Locations (13)
  • Santa Monica, California, United States
  • Miami, Florida, United States
  • Baton Rouge, Louisiana, United States
  • St Louis, Missouri, United States
  • Cincinnati, Ohio, United States
  • Cleveland, Ohio, United States
  • Hershey, Pennsylvania, United States
  • Milan, Italy
  • Padova, Italy
  • Roma, Italy
  • San Donato Milanese, Italy
  • Warsaw, Poland
  • Barcelona, Spain